Identification of genetic mutations linked to familial neurodegenerative diseases have made it possible to generate useful transgenic animal models. Studies using these transgenic animals indicate that many familial neurodegenerative diseases, such as motor neuron disease, Alzheimer's disease, prion diseases and trinucleotide repeat diseases, result from a gain of deleterious properties. The disease-specific pathology in transgenic mice demonstrates the utility of these models in elucidating pathogenic mechanisms of the disease and in developing therapeutic strategies.
Bibliographical noteFunding Information:
We thank Gopal Thinakaran, Nancy Jenkins, Neal Copeland, Don Cleveland, Carlos Pardo, and Jeffrey Rothstein for discussions of aspects of the work from our laboratory. Work cited in this review was supported by grants from the US Public Health Service (NIH NS 20471 and AG 05146) as well as the Metropolitan Life Foundation, the Adler Foundation, the American Health Assistance Foundation, the Amyotrophic Lateral Sclerosis Association, and the Alzheimer's Association. DL Price is the recipient ofa Javits Neuroscience Investigator Award (NIH NS 10580) and a Leadership & Excellence in Alzheimer's Disease (LEAD) Award (NIH AG 07914). SS Sisodia is the recipient of a Zenith Award from the Alzheimer's Association.